The international prognostic scoring system [IPSS] provides reliable risk assessment in patients with primary myelofibrosis (PMF). Recent clinical trials in PMF patients with intermediate-2 or high IPSS risk have shown a survival advantage of ruxolitinib over placebo (COMFORT-1) or best available therapy (COMFORT-2). Since crossover was allowed in these studies, we analyzed the cohort of ruxolitinib-naive patients used for developing the dynamic IPSS (DIPSS). By adopting ad hoc statistical analyses, we compared survival from diagnosis of 100 PMF patients receiving ruxolitinib within COMFORT-2 with that of 350 patients of the DIPSS study. Subjects were properly matched, and both left-truncation and right-censoring were accounted in order to compare higher IPSS risks exclusively. Patients receiving ruxolitinib had longer survival (5 years, 95% CI 2.9-7.8, versus 3.5 years, 95% CI 3.0-3.9) with a hazard ratio of 0.61 (95% CI 0.41-0.91, P=.0148). This observation suggests that ruxolitinib may modify the natural history of PMF.
Passamonti, F., Maffioli, M., Cervantes, F., Vannucchi, A., Morra, E., Barbui, T., Caramazza, D., Pieri, L., Rumi, E., Gisslinger, H., Knoops, L., Kiladjian, J.-J., Mora, B., Hollander, N., Pascutto, C., Harrison, C., & Cazzola, M. (2014). Impact of ruxolitinib on the natural history of primary myelofibrosis: a comparison of the DIPSS and the COMFORT-2 cohorts. Blood, 123(12), 1833-1835. https://doi.org/10.1182/blood-2013-12-544411 (Original work published 2014)